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Lookup NU author(s): Dr Michael GraylingORCiD, Professor James WasonORCiD
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
© 2022 The Authors. Statistics in Medicine published by John Wiley & Sons Ltd. Adapting the final sample size of a trial to the evidence accruing during the trial is a natural way to address planning uncertainty. Since the sample size is usually determined by an argument based on the power of the trial, an interim analysis raises the question of how the final sample size should be determined conditional on the accrued information. To this end, we first review and compare common approaches to estimating conditional power, which is often used in heuristic sample size recalculation rules. We then discuss the connection of heuristic sample size recalculation and optimal two-stage designs, demonstrating that the latter is the superior approach in a fully preplanned setting. Hence, unplanned design adaptations should only be conducted as reaction to trial-external new evidence, operational needs to violate the originally chosen design, or post hoc changes in the optimality criterion but not as a reaction to trial-internal data. We are able to show that commonly discussed sample size recalculation rules lead to paradoxical adaptations where an initially planned optimal design is not invariant under the adaptation rule even if the planning assumptions do not change. Finally, we propose two alternative ways of reacting to newly emerging trial-external evidence in ways that are consistent with the originally planned design to avoid such inconsistencies.
Author(s): Kunzmann K, Grayling MJ, Lee KM, Robertson DS, Rufibach K, Wason JMS
Publication type: Article
Publication status: Published
Journal: Statistics in Medicine
Year: 2022
Volume: 41
Issue: 5
Pages: 877-890
Print publication date: 28/02/2022
Online publication date: 13/01/2022
Acceptance date: 02/12/2021
Date deposited: 01/02/2022
ISSN (print): 0277-6715
ISSN (electronic): 1097-0258
Publisher: John Wiley and Sons Ltd
URL: https://doi.org/10.1002/sim.9288
DOI: 10.1002/sim.9288
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